Ocugen: FDA clears IND amendment to initiate Phase 3 clinical trial of OCU400

Ocugen announced that the U.S. FDA has cleared the company’s Investigational New Drug amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa. “The initiation of the OCU400 Phase 3 clinical trial is a significant milestone for patients with RP and a pivotal event for Ocugen as a company,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven’t had a treatment option.” Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.